Safety and Efficacy of Long-term Treatment With SCT800 in... | NCT03947567

Studies
Safety and Efficacy of Long-term Treatment With SCT800 in Previously Treated Hemophilia A Patients.

NCT03947567

Sponsor

Sinocelltech Ltd.

Status

Unknown status

Last Update Posted

Apr 28, 2020Actual

Enrollment

240Estimated

Phase

Phase 4

Conditions

Hemophilia A

Interventions

Recombinant Human Coagulation FVIII

Countries

China

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

May 2019

Overall Recruitment Status or Expanded Access Status

Unknown status

Last Known Status

Recruiting

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Jul 26, 2019Actual

Primary Completion Date

Apr 1, 2025Estimated

Completion Date

Apr 1, 2025Estimated

First Submitted Date

May 9, 2019

First Submission Date that Met QC Criteria

May 9, 2019

First Posted Date

May 13, 2019Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Apr 26, 2020

Last Update Posted Date

Apr 28, 2020Actual

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

Brief Summary

This study is a multi-center, open-label, extension trial to evaluate the safety, efficacy of SCT800 in long term prophylaxis and on-demand treatment in patients with severe hemophilia A who have been previously treated with coagulation factor VIII(FVIII) . This study includes two phases: the screening period and prophylaxis period.Prophylaxis with 25 - 50 IU/kg of SCT800 shall be administered once every other day or three times per week starting from V1, on-demand treatment is given when bleeding episodes occur. The study period is 120 consecutive weeks.

Detailed Description

Not provided

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 4

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

240Estimated

Label	Type	Description	Intervention Names
Recombinant Human Coagulation FVIII	Experimental		Drug: Recombinant Human Coagulation FVIII

Name	Type	Description	Arm Group Labels	Other Names
Recombinant Human Coagulation FVIII	Drug	Participants received SCT800 for prophylaxis and/or on-demand treatment for 120 weeks.	Recombinant Human Coagulation FVIII

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Incidence of FVIII inhibitors	The Nijmegen-Bethesda assay shall be used to monitor the production of FVIII inhibitors during the trial.	up to 120 weeks

Secondary Outcomes

Measure	Description	Time Frame
Annualized Bleeding Rate	Annualized Bleeding Rate（ABR） can be calculated using the following formula: Number of bleeding events in efficacy evaluation period/(number of days in treatment period/365.25)	up to 120 weeks
FVIII incremental in-vivo recovery

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

Male patients who are diagnosed with severe (laboratory tested FVIII:C <2%) hemophilia A;
Patients Who Completed trial of SCT800-A302 or SCT800-A 303 and New PTP(previously treated patients)(Previously received FVIII prophylactic or bleeding treatment, have the relevant records and are verified to have accumulated exposures days( EDs) ≥150 days;
Negative FVIII inhibitor assay results (laboratory tested Nijmegen-Bethesda assay result <0.6 BU(Bethesda unit)/mL);
The treatment records of at least 50EDs before screening can be obtained;
HIV negative; if HIV positive, the viral load <200 particles/uL or <400,000 copies/mL, and HIV patients must satisfy CD4+ count >200/μL;
The patient or his guardian voluntarily signed the Informed Consent Form.

Exclusion Criteria:

Known allergy to any coagulation factor VIII or any excipient; known allergy to bovine, rodent or hamster bovine;
Has a history or family history of blood coagulation factor VIII inhibitor;
Patients with other coagulation dysfunction diseases in addition to hemophilia A;
Patients with other clinically significant diseases, alcoholism, drug abuse, mental disorders or intellectual disabilities;
Patients with other severe or clinical significant diseases verified by the investigator to be unable to benefit from the clinical study;
Patients who participated in other clinical studies within one month before the first drug administration (except FVIII trials) and patients who participated in other FVIII clinical trials after signing the Informed Consent Form;

Accepts Healthy Volunteers

Sex

Male

Sex/Gender Based

Yes

Sex/Gender Description

Hemophilia A is a kind of sex chromosome recessive genetic disease and ofter occurs in male.

Minimum Age

Not provided

Maximum Age

Not provided

Standard Ages

ChildAdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Name	Role	Phone	Extension	Email
Renchi Yang	Contact	13512078851		rcyang65@163.com
Feng Xu	Contact

Overall Officials

Name	Affiliation	Role
Renchi Yang	Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College	Principal Investigator

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College	Recruiting	Tianjin			China

ID	Term
D025861	Blood Coagulation Disorders, Inherited
D001778	Blood Coagulation Disorders
D006402	Hematologic Diseases
D006425	Hemic and Lymphatic Diseases